Biomedical Catalyst – Retinal Progenitor Cell Therapy Product for Ocular Disease
Lead Research Organisation:
University College London
Department Name: UNLISTED
Abstract
Retinitis Pigmentosa (RP) is the leading cause of inherited blindness, with onset ranging from infancy to mid-thirties. Without any treatment options available, RP is severely debilitating to a patient population with many productive years of life ahead. Stem cells are recognised as offering unique therapeutic potential to treat diseases of the retina. ReNeuron owns exclusive commercialisation rights to a patented human retinal progenitor cell (hRPC) technology which offers a ground-breaking therapeutic opportunity for patients with RP to arrest on-going degeneration and regenerate sight. ReNeuron proposes to translate this opportunity through a milestone-driven and cost-effective preclinical efficacy, toxicology and safety development plan, leading to regulatory approval for a Phase I/II clinical trial in RP. ReNeuron’s hRPCs can be manufactured at scale and differentiate into photoreceptors leading to vision improvement in preclinical rodent models of degenerate retina.
Technical Summary
Retinitis Pigmentosa (RP) is the leading cause of inherited blindness, with onset ranging from infancy to mid-thirties. Without any treatment options available, RP is severely debilitating to a patient population with many productive years of life ahead. Stem cells are recognised as offering unique therapeutic potential to treat diseases of the retina. ReNeuron owns exclusive commercialisation rights to a patented human retinal progenitor cell (hRPC) technology which offers a ground-breaking therapeutic opportunity for patients with RP to arrest on-going degeneration and regenerate sight. ReNeuron proposes to translate this opportunity through a milestone-driven and cost-effective preclinical efficacy, toxicology and safety development plan, leading to regulatory approval for a Phase I/II clinical trial in RP. ReNeuron’s hRPCs can be manufactured at scale and differentiate into photoreceptors leading to vision improvement in preclinical rodent models of degenerate retina.
Organisations
People |
ORCID iD |
| Anthony Vugler (Principal Investigator) |
Publications
Semo M
(2016)
Efficacy and Safety of Human Retinal Progenitor Cells.
in Translational vision science & technology
Semo Ma'ayan
(2015)
Transplantation of human retinal progenitor cells into the P23H rat improves the scotopic ERG
in INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
| Title | Data safety monitoring board approves treatment of second patient in the clinical trial |
| Description | The second patient was successfully treated in the Phase I/II clinical trial, following a favourable outcome from the data safety monitoring board of the FDA. |
| Type | Therapeutic Intervention - Cellular and gene therapies |
| Current Stage Of Development | Early clinical assessment |
| Year Development Stage Completed | 2016 |
| Development Status | Under active development/distribution |
| Clinical Trial? | Yes |
| Impact | This safety data has attracted further investor interest in the ReNeurons hRPC product and the companies programme in retinitis pigmentosa. |
| URL | http://4965zs3ha2l125fk78zkozo3.wpengine.netdna-cdn.com/wp-content/uploads/ReNeuron-June-2016-retina... |
| Title | First patient treated in the clinical trial |
| Description | The first patient with advanced retinitis pigmentosa was treated with ReNeuron's hRPC product in the Phase I/II US clinical trial. This marks the first US clinical trial activity by the British company ReNeuron. This achievement was made possible by our preclinical work on this Innovate UK / MRC funded grant. |
| Type | Therapeutic Intervention - Cellular and gene therapies |
| Current Stage Of Development | Early clinical assessment |
| Year Development Stage Completed | 2016 |
| Development Status | Under active development/distribution |
| Clinical Trial? | Yes |
| Impact | Yes, the preclinical work at UCL indicated that the hRPC cell line may have utility in treating other degenerative retinal diseases. This has led to a new proof of concept experiment in my laboratory at UCL, funded by ReNeuron. |
| URL | http://4965zs3ha2l125fk78zkozo3.wpengine.netdna-cdn.com/wp-content/uploads/ReNeuron-March-2016-first... |
| Title | Phase II clinical trial |
| Description | Following a successful Phase I trial, with three patient groups treated with ascending doses of the hRPC product (total of 9 patients with retinitis Pigments (RP)), the Data Safety Monitoring Board has given approval for the study to progress into its phase II element. This will involve the recruitment and treatment of a further 6 RP patients with less advanced retinal degeneration using the highest cell dose defined in the phase I trial. |
| Type | Therapeutic Intervention - Cellular and gene therapies |
| Current Stage Of Development | Early clinical assessment |
| Year Development Stage Completed | 2017 |
| Development Status | Under active development/distribution |
| Clinical Trial? | Yes |
| Impact | The development process has seen a move from a freshly-prepared to frozen cell product. This new frozen cell product is an important innovation, which will facilitate future upscaling of the treatment for application in a Phase III clinical trial and cell manufacture to meet patient demand. |
| URL | http://4965zs3ha2l125fk78zkozo3.wpengine.netdna-cdn.com/wp-content/uploads/ReNeuron-hRPC-update-Nov-... |
| Title | Retinal progenitor therapy product for ocular disease |
| Description | The medical product is a human retinal progenitor cell line generated by the company ReNeuron which is in an active stage of pre-clinical development. I have completed several pre-clinical efficacy studies so far during the project and have also assisted in the completion of a large scale pre-clinical safety study. These studies have now been compiled into pre-IND documents in preparation for the pre-IND meeting with the FDA in the US later this month (November 2014). This development has been funded by MRC through the Technology Strategy Board. |
| Type | Therapeutic Intervention - Cellular and gene therapies |
| Current Stage Of Development | Refinement. Non-clinical |
| Year Development Stage Completed | 2014 |
| Development Status | Under active development/distribution |
| Impact | The development process has involved pre-clinical studies using rodent models of retinal disease. The data from these studies will result in multiple scientific publications (anticipated following completion of the project in March 2015). |
| Title | Safety and Tolerability of hRPC in Retinitis Pigmentosa (hRPCRP) |
| Description | Following a successful IND application to the FDA, ReNeuron have now initiated a Phase I/II clinical trial in patients with advanced Retinitis Pigmentosa. This is a dose-escalation study with will assess the safety and tolerability of their hRPC product in humans. This comes as a direct result of my technology strategy board (Innovate UK, TSB) funded research with Reneuron, during which I designed and implemented preclinical safety and efficacy studies and compiled regulatory documents for the IND submission. |
| Type | Therapeutic Intervention - Cellular and gene therapies |
| Current Stage Of Development | Early clinical assessment |
| Year Development Stage Completed | 2016 |
| Development Status | Under active development/distribution |
| Clinical Trial? | Yes |
| Impact | During the pre-clinical efficacy testing, we were able to fully phenotype the hRPCs and insights from this work have now opened up new research / exploitation avenues for the ReNeuron product. On the basis of these findings, ReNeuron put in another application to Innovate UK (with myself) to test the efficacy of hRPCs in other preclinical models of retinal disease. |
| URL | https://clinicaltrials.gov/ct2/show/NCT02464436?term=Reneuron&rank=1 |
| Description | Invited talk at the International Society of Cellular Therapy |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | This was a talk at an international conference describing my collaboration with ReNeuron and progress towards the treatment of patients with the hRPC drug product. The talk was well received and sparked a number of questions afterwards. |
| Year(s) Of Engagement Activity | 2016 |
| URL | http://c.ymcdn.com/sites/www.celltherapysociety.org/resource/resmgr/2016na_regmtgpresentations/ISCTN... |
| Description | Seminar to students and staff at UCL Institute of Ophthalmology "Translating a cell-based therapy for retinal disease" |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Postgraduate students |
| Results and Impact | This was a 60 minute "masterclass" seminar open to the whole of UCL during which I described my work relating to cellular therapy. In particular I focused on the pathway which needs to be followed in order to obtain regulatory approval to commence a phase I/II clinical trial for retinal disease. The seminar was well-attended (over 50 students and academic staff) and I continued an open discussion with a group of students for another hour after the seminar. During this post-seminar session, I posed them questions relating to my TSB funded work with ReNeuron and they discussed these points in groups and then responded to me, posing their own questions in return. |
| Year(s) Of Engagement Activity | 2016 |
| URL | https://www.ucl.ac.uk/brain-sciences/student-news/ophthalmology-seminar-series |